CO2018004564A2 - Genes de ataxia de friedreich modificados y vectores para terapia génica - Google Patents

Genes de ataxia de friedreich modificados y vectores para terapia génica

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Publication number
CO2018004564A2
CO2018004564A2 CONC2018/0004564A CO2018004564A CO2018004564A2 CO 2018004564 A2 CO2018004564 A2 CO 2018004564A2 CO 2018004564 A CO2018004564 A CO 2018004564A CO 2018004564 A2 CO2018004564 A2 CO 2018004564A2
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CO
Colombia
Prior art keywords
vectors
gene therapy
friedreich ataxia
modified
genes
Prior art date
Application number
CONC2018/0004564A
Other languages
English (en)
Inventor
Richard J Samulski
Original Assignee
Bamboo Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Bamboo Therapeutics Inc filed Critical Bamboo Therapeutics Inc
Publication of CO2018004564A2 publication Critical patent/CO2018004564A2/es

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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Neurosurgery (AREA)
  • General Engineering & Computer Science (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Biophysics (AREA)
  • Mycology (AREA)
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  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Plant Pathology (AREA)

Abstract

La presente invención se relaciona con genes y vectores modificados de FXN que comprende el gen modificado que proporciona una expresión incrementada de la proteína de frataxina codificada que puede utilizarse para el tratamiento de la ataxia de Friedreich.
CONC2018/0004564A 2015-11-05 2018-04-27 Genes de ataxia de friedreich modificados y vectores para terapia génica CO2018004564A2 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562251288P 2015-11-05 2015-11-05
US201662411980P 2016-10-24 2016-10-24
PCT/IB2016/056572 WO2017077451A1 (en) 2015-11-05 2016-11-01 Modified friedreich ataxia genes and vectors for gene therapy

Publications (1)

Publication Number Publication Date
CO2018004564A2 true CO2018004564A2 (es) 2018-12-14

Family

ID=57288476

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CONC2018/0004564A CO2018004564A2 (es) 2015-11-05 2018-04-27 Genes de ataxia de friedreich modificados y vectores para terapia génica

Country Status (18)

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US (2) US10548947B2 (es)
EP (1) EP3370778A1 (es)
JP (2) JP6767483B2 (es)
KR (2) KR102200642B1 (es)
CN (1) CN108348621A (es)
AU (2) AU2016347887B2 (es)
BR (1) BR112018007453A2 (es)
CA (1) CA2947584A1 (es)
CO (1) CO2018004564A2 (es)
HK (1) HK1250924A1 (es)
IL (1) IL259141A (es)
MX (1) MX2018005084A (es)
PE (1) PE20181156A1 (es)
PH (1) PH12018500964A1 (es)
RU (2) RU2021102893A (es)
SG (2) SG11201802408RA (es)
TW (2) TWI654301B (es)
WO (1) WO2017077451A1 (es)

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TWI654301B (zh) 2019-03-21
TWI743442B (zh) 2021-10-21
RU2018116076A3 (es) 2020-08-21
TW201723179A (zh) 2017-07-01
WO2017077451A1 (en) 2017-05-11
JP2018531609A (ja) 2018-11-01
MX2018005084A (es) 2019-05-16
BR112018007453A2 (pt) 2018-10-23
KR20180077275A (ko) 2018-07-06
CN108348621A (zh) 2018-07-31
JP6767483B2 (ja) 2020-10-14
SG10201912763QA (en) 2020-02-27
AU2020200976B2 (en) 2021-09-30
HK1250924A1 (zh) 2019-01-18
RU2743792C2 (ru) 2021-02-26
AU2016347887B2 (en) 2019-11-14
EP3370778A1 (en) 2018-09-12
KR20210005994A (ko) 2021-01-15
AU2020200976A1 (en) 2020-02-27
TW201920673A (zh) 2019-06-01
JP2021019591A (ja) 2021-02-18
RU2018116076A (ru) 2019-10-28
RU2021102893A (ru) 2021-03-03
AU2016347887A1 (en) 2018-04-19
US10548947B2 (en) 2020-02-04
IL259141A (en) 2018-06-28
PH12018500964A1 (en) 2018-11-12
PE20181156A1 (es) 2018-07-19
KR102200642B1 (ko) 2021-01-12
US20170128528A1 (en) 2017-05-11
CA2947584A1 (en) 2017-05-05
US20200384073A1 (en) 2020-12-10
SG11201802408RA (en) 2018-05-30

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